An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03675126|
Recruitment Status : Recruiting
First Posted : September 18, 2018
Last Update Posted : January 23, 2019
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: SRP-5051||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051|
|Actual Study Start Date :||December 19, 2018|
|Estimated Primary Completion Date :||November 1, 2020|
|Estimated Study Completion Date :||November 1, 2020|
Participants to receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 (NCT03375255) study.
SRP-5051 administered as an IV infusion.
- Number of participants with adverse events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]An AE is any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug, whether or not considered related to the study drug.
- Maximum plasma concentration (Cmax) of SRP-5051 [ Time Frame: End of infusion ]Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
- Area under the plasma concentration versus time curve (AUC) of SRP-5051 [ Time Frame: Pre-dose, end of infusion, 6 hours post-dose ]Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
- Number of participants with clinically relevant abnormalities, as assessed by vital sign measurements, physical examination findings, clinical laboratory tests and electrocardiograms (ECGs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]*A clinically relevant abnormality is an abnormality confirmed by repeat testing that is changed sufficiently from screening/baseline so that, in the judgment of the Investigator, a change in management is warranted.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03675126
|Contact: Medical information||+1 888 727 firstname.lastname@example.org|
|United States, Florida|
|NW FL Clinical Research Group, LLC||Recruiting|
|Gulf Breeze, Florida, United States, 32561|
|Contact: Shae Colbert 850-934-1299 email@example.com|
|Principal Investigator: Weldon Mauney, MD|
|United States, Georgia|
|Rare Disease Research, LLC||Recruiting|
|Atlanta, Georgia, United States, 30318|
|Contact: Han C Phan, MD 678-883-6897 firstname.lastname@example.org|
|Principal Investigator: Han C Phan, MD|
|United States, Kansas|
|University of Kansas Medical Center||Recruiting|
|Kansas City, Kansas, United States, 66160|
|Contact: Katherine Roath 913-945-9928 email@example.com|
|Principal Investigator: Jeffrey Statland, MD|
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of UPMC||Recruiting|
|Pittsburgh, Pennsylvania, United States, 15224|
|Contact: Jennifer Monahan 412-692-5176 firstname.lastname@example.org|
|Principal Investigator: Hoda Abdel-Hamid, MD|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|